About this episode

This episode discusses how a rare diagnosis led to a global scientific effort for PACS2 therapy.

What happens when parents refuse to accept a one percent chance? In this episode of Sounds of Science , Piotr and Gosia Kosla share how their daughter Lena’s ultra‑rare diagnosis sparked a global scientific effort, advancing a potential treatment from discovery to an active preclinical toxicology study in just two years. Show Notes General Toxicology | Charles River Preclinical GLP Toxicology Studies Painting a Picture of Hope to Treat PACS2 | Podcast PACS2 Research Foundation

People in this episode

Host: Charles River

Guests: Piotr Kosla, Gosia Kosla

Topics covered

• PACS2 therapy
• toxicology
• rare diseases
• scientific research
• preclinical studies

Keywords

• PACS2
• toxicology
• rare diagnosis
• preclinical study
• scientific effort

Mentioned in this episode

Organizations: PACS2 Research Foundation, Charles River

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